Prolonged activity of the transposase helper may raise safety concerns during DNA transposon-based gene therapy

Prolonged activity of the transposase helper may raise safety concerns during DNA transposon-based gene therapy

DNA transposon-based gene delivery vectors represent a promising new branch of randomly integrating vector development for gene therapy. For the side-by-side evaluation of the piggyBac and Sleeping Beauty systems—the only DNA transposons currently employed in clinical trials—during therapeutic inter...

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Bibliographic Details
Main Authors: Gergely Imre, Bertalan Takács, Erik Czipa, Andrea Bakné Drubi, Gábor Jaksa, Dóra Latinovics, Andrea Nagy, Réka Karkas, Liza Hudoba, Bálint Márk Vásárhelyi, Gabriella Pankotai-Bodó, András Blastyák, Zoltán Hegedűs, Péter Germán, Balázs Bálint, Khaldoon Sadiq Ahmed Abdullah, Anna Georgina Kopasz, Anita Kovács, László G. Nagy, Farkas Sükösd, Lajos Pintér, Thomas Rülicke, Endre Barta, István Nagy, Lajos Haracska, Lajos Mátés
Format: Article
Language:English
Published: Elsevier 2023-06-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
tyrosinemia type I
gene therapy
DNA transposon
Sleeping Beauty
piggyBac
prolonged transpositional activity
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050123000396

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http://www.sciencedirect.com/science/article/pii/S2329050123000396

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